Page 59 - CIBERER2016-ENG

- Cell therapy aimed at enhancing the therapeutic effect of mesenchymal stem cells through its phenotypic modification.
- Manufacture of clinical grade stromal mesenchymal cells.
During 2016 our activity has been focused on the development of research projects of the National Plan for Research and the 7th Framework Programme of the EU (FAMOCURE, EPISEVI 2.0, PKRESET and EUROFANCOLEN Project). The work is done in collaboration with other groups from the CIBER, including Associated Clinical Groups (i.e. H. Niño Jesús GCV19 y H San Joan de Deu GCV18) and the Hospital Fundación Jimenez Diaz (with whom we form a Joint Unit for Advanced Therapies). Additionally, we offer collaboration with other researchers from the CIBER to develop new advanced therapies for rare diseases.
Most relevant scientific articles
• Lopez-Santalla M., Mancheno-Corvo P., Menta R., Lopez-Belmonte J., Delarosa O., Bueren J.A. et al. Human Adipose-Derived Mesenchymal Stem Cells Modulate Experimental Autoimmune Arthritis by Modifying Early Adaptive T Cell Responses. Stem Cells. 2016;33(12):3493-3503.
• Pulecio J., Alejo-Valle O., Capellera-Garcia S., Vitaloni M., Rio P., Mejia-Ramirez E. et al. Direct Conversion of Fibroblasts to Megakaryocyte Progenitors. Cell Reports. 2016;17(3):671-683.
• Leon-Rico D., Aldea M., Sanchez-Baltasar R., Mesa-Nunez C., Record J., Burns S.O. et al. Lentiviral Vector- Mediated Correction of a Mouse Model of Leukocyte Adhesion Deficiency Type i. Human Gene Therapy. 2016;27(9):668-678.
• Garcia-Gomez M., Calabria A., Garcia-Bravo M., Benedicenti F., Kosinski P., Lopez-Manzaneda S. et al. Safe and efficient gene therapy for pyruvate kinase deficiency. Molecular Therapy. 2016;24(7):1187-1198.
• Lopez-Luque J., Caballero-Diaz D., Martinez-Palacian A., Roncero C., Moreno-Caceres J., Garcia-Bravo M. et al. Dissecting the role of epidermal growth factor receptor catalytic activity during liver regeneration and hepatocarcinogenesis. Hepatology. 2016;63(2):604-619.
Hightlights
As a complement to the National Programs and programs of the European Commission, during 2016
three lentiviral vectors developed by our laboratory have been licensed to a US company, Rocket-Pharma. Additionally, a contract has been signed for the development with Rocket Pharma for the development of a clinical trial in patients with erythrocyte pyruvate kinase deficiency, and also a Framework Agreement between this company and the CIEMAT, CIBER and Fundación Jiménez Díaz.
In the field of gene therapy, our work continues focused on the research and gene therapy of rare diseases that affect blood cells. These include congenital bone marrow failures, as well as congenital anemias and primary immunodeficiencies. Particularly throughout 2016 we have made significant progresses in the following fields.
• Implementation of the first clinical trial of patients with Fanconi anemia of subtype A by correction with
lentiviral vectors of mobilized peripheral blood cells.
• Designation of two orphan drugs in the European Commision and the Food and Drug Agency in the USA for
the treatment of erythrocyte pyruvate kinase deficiency and type I leukocyte adhesion deficiency.
• Development of gene editing strategies in hematopoietic stem cells for Fanconi anemia and deficiency in
erythrocyte pyruvate kinase.
• Reprogramming and gene therapy of cells from patients with primary hyperoxaluria type 1.
In the field of the studies with mesenchymal stromal cells (MSC), we have worked on the following research lines:
• Characterization of a new application of MSCs to facilitate the engraftment of genetically corrected hematopoietic stem cells.
• Preclinical demonstration of the therapeutic effect of MSCs in rheumatoid arthritis and inflammatory bowel disease.
• Phenotypic modification of MSCs to enhance their therapeutic effect.
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research groups 59